A Real-world Study of Disease-modifying Therapy Treatment Outcomes in Patients With Spinal Muscular Atrophy

Part of paid clinical trials in East Hanover, New Jersey.

Sponsor
Novartis Pharmaceuticals
Study ID
NCT07403214
Status
Completed

Conditions

  • Muscular Atrophy, Spinal

Eligibility Criteria

Sex
ALL
Age
N/A - N/A
Healthy Volunteers
Not accepted

Study Details

The aim of this study was to evaluate treatment patterns, SMA-related complications and services, and all-cause medical encounters of patients with SMA receiving multiple DMTs in the real world. The DMTs included onasemnogene abeparvovec-xioi (OA), nusinersen, and risdiplam. This study was conducted using data from the Komodo Health Research Database (KRD+) between 01 January 2016 and 31 October 2024.

Key Dates

Start date
Jun 4, 2025
Status verified
Feb 2026
Primary completion
Jun 30, 2025
Completion
Jun 30, 2025

Study Design

Enrollment
4,805 participants (actual)

Arms

  • Arm: DMT Cohort A
    Patients with SMA who were treated with at least 1 DMT and had continuous health insurance coverage in KRD+ closed claims at the time of first DMT initiation.
  • Arm: DMT Cohort B
    Patients with SMA who were treated with at least 1 DMT and received the first DMT at age 2 or younger.
  • Arm: DMT Cohort B1
    A sub-group of DMT Cohort B who started the first DMT between 2016 and 2021.
  • Arm: DMT Cohort B2
    A sub-group of DMT Cohort B who started the first DMT between 2022 and 2024.
  • Arm: DMT Cohort C
    Patients with SMA types 1-3 who were treated with multiple DMTs.
  • Arm: DMT Cohort D
    A subset of DMT Cohort C who received the first DMT at age two or younger.
  • Arm: DMT Cohort D1
    A sub-group of DMT Cohort D who started the first DMT between 2016 and 2021.
  • Arm: DMT Cohort D2
    A sub-group of DMT Cohort D who started the first DMT between 2022 and 2024.

Primary Outcome Measure

Number of Patients by SMA-related Complication, Procedure, and Durable Medical Equipment (DME) Before Receiving the First DMT (Period I) [ Time Frame: Up to approximately 1 year ]

Locations (1)

FacilityCityStateZIPSite coordinators
NovartisEast HanoverNew Jersey07936-

Find similar trials in East Hanover, NJ

By research site
Novartis· East Hanover, NJ

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